JP5829372B2 - 神経代謝性疾患のための遺伝子治療 - Google Patents

神経代謝性疾患のための遺伝子治療 Download PDF

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JP5829372B2
JP5829372B2 JP2008510224A JP2008510224A JP5829372B2 JP 5829372 B2 JP5829372 B2 JP 5829372B2 JP 2008510224 A JP2008510224 A JP 2008510224A JP 2008510224 A JP2008510224 A JP 2008510224A JP 5829372 B2 JP5829372 B2 JP 5829372B2
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aav
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mice
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JP2008540445A5 (enExample
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マルコ・エイ・パッシーニ
ジェイムズ・ダッジ
グレゴリー・アール・スチュアート
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JP2008510224A 2005-05-02 2006-05-02 神経代謝性疾患のための遺伝子治療 Expired - Lifetime JP5829372B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US67705705P 2005-05-02 2005-05-02
US60/677,057 2005-05-02
US68580805P 2005-05-31 2005-05-31
US60/685,808 2005-05-31
PCT/US2006/017242 WO2006119458A1 (en) 2005-05-02 2006-05-02 Gene therapy for neurometabolic disorders

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JP2013201138A Division JP2014037418A (ja) 2005-05-02 2013-09-27 神経代謝性疾患のための遺伝子治療

Publications (3)

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JP2008540445A JP2008540445A (ja) 2008-11-20
JP2008540445A5 JP2008540445A5 (enExample) 2009-07-09
JP5829372B2 true JP5829372B2 (ja) 2015-12-09

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JP2008510224A Expired - Lifetime JP5829372B2 (ja) 2005-05-02 2006-05-02 神経代謝性疾患のための遺伝子治療
JP2013201138A Pending JP2014037418A (ja) 2005-05-02 2013-09-27 神経代謝性疾患のための遺伝子治療
JP2015197051A Expired - Lifetime JP6338560B2 (ja) 2005-05-02 2015-10-02 神経代謝性疾患のための遺伝子治療

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JP2013201138A Pending JP2014037418A (ja) 2005-05-02 2013-09-27 神経代謝性疾患のための遺伝子治療
JP2015197051A Expired - Lifetime JP6338560B2 (ja) 2005-05-02 2015-10-02 神経代謝性疾患のための遺伝子治療

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US (2) US10632213B2 (enExample)
EP (4) EP1879624B1 (enExample)
JP (3) JP5829372B2 (enExample)
CN (4) CN104306986A (enExample)
AT (1) ATE525092T1 (enExample)
AU (1) AU2006243776A1 (enExample)
BR (1) BRPI0611379A2 (enExample)
CA (2) CA2607173C (enExample)
ES (1) ES2887076T3 (enExample)
IL (2) IL187078A (enExample)
PL (4) PL2420256T3 (enExample)
PT (4) PT3520823T (enExample)
WO (1) WO2006119458A1 (enExample)

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EP3520823A1 (en) 2019-08-07
CA2607173C (en) 2018-04-24
CA2998603C (en) 2019-07-23
CA2607173A1 (en) 2006-11-09
ATE525092T1 (de) 2011-10-15
EP1879624B1 (en) 2011-09-21
US11957765B2 (en) 2024-04-16
EP1879624A1 (en) 2008-01-23
PL3520823T3 (pl) 2021-11-22
EP1879624A4 (en) 2009-03-25
IL187078A (en) 2015-06-30
EP2420256B1 (en) 2016-06-22
CN107362371A (zh) 2017-11-21
PL1879624T3 (pl) 2012-02-29
CN101212988A (zh) 2008-07-02
JP6338560B2 (ja) 2018-06-06
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